JUPITER, Fla., Aug. 22, 2018 /PRNewswire/ -- Jupiter Orphan Therapeutics, Inc. ("JOT"), Jupiter, FL, today announced that it has received new data regarding an increase in mitochondrial biogenesis in liver and brain from a recently performed study in a frequently studied Alzheimer's disease animal model, the triple transgenic (APPsw / PS1M146V / TauP301L) mouse.
Daily oral treatment, 50mg/kg, of mice for 5 weeks resulted in a marked increase of liver (72%) and brain (30%) mitochondrial DNA in JOTROL™ treated mice compared to vehicle controls. JOTROL™ induced similar increases of the mitochondrial genes Nd1 and Rnr1 and no change in the nuclear gene NEB. Our results with JOTROL™ are in line with a growing body of literature showing that select polyphenolic compounds, notably high doses of resveratrol, exert direct effects on mitochondrial ultra-structure and function. In other words, treatment with JOTROL™ will increase the effectiveness of the cell's mitochondrial machinery. Resveratrol, the active component of JOTROL™, is in fact sometimes referred to as a mitochondrial "nutrient."
"This is a major milestone in JOT's relatively short history and marks an important value inflection point. Although Friedreich's ataxia, JOT's lead targeted indication, is to a high degree a mitochondrial disease, we originally did not grasp the unique effectiveness of our JOTROL product in the overall mitochondrial disease market until we received these results. This increases our earlier targeted market of $500 Million for JOTROL to over $5 billion, in USA alone," said Chief Executive Officer, Christer Rosén of JOT.
"I am not aware of any other safe drug candidates that induce robust mitochondrial biogenesis, let alone in the brain. Another big plus is that JOTROL will be an oral medication, which patients prefer, so no infusions or injections," stated CMO, Claes Wahlestedt, MD PhD.
"Increased mitochondrial biogenesis is a promising approach to treating some forms of mitochondrial diseases, said Philip Yeske, Science and Alliance Officer for the United Mitochondrial Disease Foundation. The mitochondrial disease community looks forward to further therapeutic development efforts by JOT in follow-up to the preliminary pre-clinical work reported on mice."
"This is a very significant increase in mitochondrial numbers and I expect it to have an impact on health in patients with mitochondrial disease," stated Professor Gino Cortopassi, UC Davis.
JOT has developed a unique patented trans-resveratrol product called JOTROL™. JOTROL™ is expected to deliver the high amount of resveratrol in blood plasma that is required to achieve therapeutic effects. JOTROL™ remedies the scientifically well documented poor bioavailability and dose limiting side effects associated with resveratrol, allowing delivery of therapeutic, safe doses of resveratrol without debilitating GI-side effects.
Mitochondrial diseases result from failures of the mitochondria, specialized compartments present in every cell of the body (except red blood cells).
Mitochondria are responsible for creating more than 90% of the energy needed by the body to sustain life and support organ function. When they fail, less and less energy is generated within the cell. Cell injury and even cell death follow. If this process is repeated throughout the body, whole organ systems begin to fail.
The parts of the body, such as the heart, brain, muscles and lungs, requiring the greatest amounts of energy are the most affected. Mitochondrial disease is difficult to diagnose, because it affects each individual differently. Symptoms can include seizures, strokes, severe developmental delays, inability to walk, talk, see, and digest food combined with a host of other complications. If three or more organ systems are involved, mitochondrial disease should be suspected. Although mitochondrial disease primarily affects children, adult onset is becoming more common.
Mitochondrial defects are a central factor in human health and disease
Mitochondrial dysfunction is at the core of a surprising range of very common illnesses and conditions, and a promising new avenue for their treatment. As the mitochondria are responsible for producing energy, any illness that has an energy problem could be related to the mitochondria.
Please visit www.umdf.org for details of rare mitochondrial diseases. Additional diseases in which mitochondrial dysfunction have been implicated include:
Alzheimer's Dementia, Parkinson's disease, Huntington Disease, Amyotrophic Lateral Sclerosis (ALS), mental retardation, deafness and blindness, diabetes, obesity, cardiovascular disease and stroke. Over 50 million people in the US suffer from these chronic degenerative disorders. While it cannot yet be said that mitochondrial defects cause these problems, it is clear that mitochondria are involved because their function is measurably disturbed.
Even autoimmune diseases such as multiple sclerosis, Sjogrens syndrome, lupus and rheumatoid arthritis appear to have a mitochondrial basis to illness.
Mitochondrial dysfunction has been associated with a wide range of solid tumors, proposed to be central to the aging process, and found to be a common factor in the toxicity of a variety of physical and chemical agents.
About Friedreich's Ataxia (FA)
Friedreich's Ataxia (FA) is a rare inherited disease that causes damage to the nervous system as well as diminished mobility. FA usually begins in childhood and leads to impaired muscle coordination (ataxia) which worsens over time. It is caused by a defect (mutation) in the Frataxin (FXN) gene. Friedreich's ataxia is recessive, meaning it only occurs in someone who inherits two defective copies of the gene, one from each parent. Although rare, FA in the most common form of hereditary ataxia, affecting about 1 in 50,000 people in the United States. There are no approved treatments available today. Visit the FARA (Friedreich's Ataxia Research Alliance) website, www.curefa.org, for further details on this rare disease. Murdoch Children's Research Institute, a JOT partner, has conducted an open label study showing that high dose of resveratrol (not JOTROL), with associated GI-side effects, had significant positive impact in 4 vital endpoints. Target, for this indication, is to reproduce this study with JOTROL and reproduce the same positive results without side effects.
About Jupiter Orphan Therapeutics
Jupiter Orphan Therapeutics, Inc. (JOT) is a clinical stage specialty pharmaceutical company developing therapies for rare diseases. JOT, a Delaware Corporation with its principal office located in Jupiter, FL, USA, was founded in the summer of 2015. In its short period of operations, JOT has assembled a very strong management and scientific team, developed JOTROL™ as a platform product to treat multiple rare diseases as well as collaborating with established partners in other disease areas.